Gene therapy for haemophilia: The end of a 'royal pathology' in the third millennium?

Haemophilia

Volumn 7, Issue 5, 2001, Pages 441-445

  Liras, A ^a  

^a UNIVERSIDAD AUTÓNOMA DE MADRID   (Spain)

Author keywords

FIX; FVIII; Gene therapy; Haemophilia; Viral vectors

Indexed keywords

BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 9; VIRUS VECTOR;

ADENO ASSOCIATED VIRUS; ADENOVIRUS; ANIMAL EXPERIMENT; ANIMAL MODEL; CLINICAL STUDY; CLINICAL TRIAL; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; DRUG APPROVAL; FOOD AND DRUG ADMINISTRATION; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER; HEMOPHILIA; HEMOPHILIA A; HEMOPHILIA B; HUMAN; MOUSE; NONHUMAN; PRIORITY JOURNAL; RETROVIRUS; REVIEW; SIDE EFFECT; VIRUS RECOMBINANT;

ANIMALS; FACTOR IX; FACTOR VIII; FORECASTING; GENE THERAPY; HEMOPHILIA A; HUMANS; TRANSDUCTION, GENETIC;

EID: 0034804403     PISSN: 13518216     EISSN: None     Source Type: Journal    
DOI: 10.1046/j.1365-2516.2001.00554.x     Document Type: Review

References (17)

1. Targeted disruption of the mouse factor VIII gene produces a model of haemophilia A
2. A coagulation factor IX-deficient mouse model for human hemophilia B
3. The canine factor VIII cDNA and 5′ flanking sequence
4. Canine hemophilia B resulting from a point mutation with unusual consequences
5. Adenovirus-mediated expression of human coagulation factor IX in the Rhesus macaque is associated with dose-limiting toxicity
6. Transient depletion of CD4 lymphocyte improves efficacy of repeated administration of recombinant adenovirus in the ornithine transcarbamylase-deficient sparse fur mouse
7. Intravenous administration of an E1/E3-deleted adenoviral vector induces tolerance to factor IX in C57BL/6 mice
8. Prolonged transgene expression mediated by a helper-dependent adenoviral vector (hdAd) in the central nervous system
9. Long-term expression of human coagulation factor VIII and correction of hemophilia A after in vivo retroviral gene transfer in factor VIII-deficient mice
10. Sustained high-level expression of full-length human factor VIII and restoration of clotting activity in hemophilic mice using a minimal adenovirus vector
11. In vivo evaluation of an adenoviral vector encoding canine factor VIII. High-level, sustained expression in hemophiliac mice
12. Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector
13. Persistent expression of canine factor IX in hemophilia B dogs
14. Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors
15. Sustained correction of bleeding disorder in hemophilia B mice by gene therapy
16. Improved muscle-derived expression of human coagulation factor IX from a skeletal actin/CMV hybrid enhancer/promoter
17. Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector